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Clinical experience with tovorafenib in adults with treatment-refractory high- and low-grade gliomas
Maria Ioannou1,2, Setu Mehta1, Anirudra Devkota2
1Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Background:
BRAF alterations are common in pediatric low-grade gliomas (LGG) and a subset of high-grade gliomas (HGG) in adults and children. BRAF-targeted therapy can be effective at preventing tumor growth, though resistance commonly emerges in HGG. Recently, tovorafenib was FDA approved for recurrent or refractory LGG with BRAFV600 alterations or BRAF rearrangements. While the trial showed it is effective for children with LGG previously treated with BRAF or MEK inhibitors, the safety in adults and efficacy in HGG is unknown.
Methods:
A cohort of appropriate patients was identified through routine clinical care. This research was conducted in accordance with IRB regulations with a waiver of written consent.
Results:
Seven adults (5 HGG, 2 LGG) who received tovorafenib were identified. All patients had received prior BRAF-targeted therapy, and all patients with HGG had received prior radiation and temozolomide as well. Three individuals (2 HGG, 1 LGG) experienced stable disease or better for 4 or more months. Median duration of treatment was 8 weeks (range 3 weeks to 10 months). Two individuals experienced CTCAE grade 4 intratumoral hemorrhage (1 HGG, 1 LGG). Two patients remain on therapy at 4 and 10 months of treatment (1 HGG, 1 LGG).
Conclusion:
Our experience with tovorafenib indicates some limited efficacy in HGG in combination with other standard treatments. These observations demonstrate the need for further clinical trials in patients with HGG to understand potential clinical utility, either earlier in the disease course or in combination with other therapies.
Insights
Tovorafenib shows limited efficacy in adult high-grade gliomas (HGG), necessitating further clinical trials. This BRAF-targeted therapy requires more research for potential utility in HGG treatment.
Area of Science:
- Neuro-oncology
- Pharmacology
- Clinical Trials
Background:
- BRAF alterations are prevalent in pediatric low-grade gliomas (LGG) and adult/pediatric high-grade gliomas (HGG).
- BRAF-targeted therapy, including recently FDA-approved tovorafenib for pediatric LGG, shows efficacy but resistance is common in HGG.
- The safety and efficacy of tovorafenib in adult HGG remain largely unknown.
Purpose of the Study:
- To evaluate the safety and efficacy of tovorafenib in adult patients with high-grade gliomas (HGG) and low-grade gliomas (LGG).
- To assess the clinical utility of tovorafenib in adult HGG, considering prior treatments.
Main Methods:
- Retrospective review of seven adult patients (5 HGG, 2 LGG) treated with tovorafenib.
- Patients had received prior BRAF-targeted therapy; HGG patients also received radiation and temozolomide.
- Data collected on treatment duration, response (stable disease or better), and adverse events.
Main Results:
- Median treatment duration was 8 weeks; two patients remain on therapy at 4 and 10 months.
- Three patients (2 HGG, 1 LGG) achieved stable disease or better for over 4 months.
- Two patients experienced grade 4 intratumoral hemorrhage.
Conclusions:
- Tovorafenib demonstrates limited efficacy in adult HGG when used with standard treatments.
- Further clinical trials are essential to determine the potential utility of tovorafenib in HGG, possibly earlier in treatment or in combination therapies.
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